Blockade of nonhormonal fibroblast growth factors by FP-1039 inhibits growth of multiple types of cancer

Sci Transl Med. 2013 Mar 27;5(178):178ra39. doi: 10.1126/scitranslmed.3005414.

Abstract

The fibroblast growth factor (FGF) pathway promotes tumor growth and angiogenesis in many solid tumors. Although there has long been interest in FGF pathway inhibitors, development has been complicated: An effective FGF inhibitor must block the activity of multiple mitogenic FGF ligands but must spare the metabolic hormone FGFs (FGF-19, FGF-21, and FGF-23) to avoid unacceptable toxicity. To achieve these design requirements, we engineered a soluble FGF receptor 1 Fc fusion protein, FP-1039. FP-1039 binds tightly to all of the mitogenic FGF ligands, inhibits FGF-stimulated cell proliferation in vitro, blocks FGF- and vascular endothelial growth factor (VEGF)-induced angiogenesis in vivo, and inhibits in vivo growth of a broad range of tumor types. FP-1039 antitumor response is positively correlated with RNA levels of FGF2, FGF18, FGFR1c, FGFR3c, and ETV4; models with genetic aberrations in the FGF pathway, including FGFR1-amplified lung cancer and FGFR2-mutated endometrial cancer, are particularly sensitive to FP-1039-mediated tumor inhibition. FP-1039 does not appreciably bind the hormonal FGFs, because these ligands require a cell surface co-receptor, klotho or β-klotho, for high-affinity binding and signaling. Serum calcium and phosphate levels, which are regulated by FGF-23, are not altered by administration of FP-1039. By selectively blocking nonhormonal FGFs, FP-1039 treatment confers antitumor efficacy without the toxicities associated with other FGF pathway inhibitors.

MeSH terms

  • Calcium / blood
  • Fibroblast Growth Factor-23
  • Fibroblast Growth Factors / antagonists & inhibitors*
  • Fibroblast Growth Factors / metabolism
  • Humans
  • Immunoglobulin G / therapeutic use*
  • Neoplasms / drug therapy*
  • Neoplasms / metabolism*
  • Oncogene Proteins, Fusion / therapeutic use*
  • Phosphates / blood
  • Receptor, Fibroblast Growth Factor, Type 1 / therapeutic use*
  • Recombinant Fusion Proteins

Substances

  • FGF23 protein, human
  • Immunoglobulin G
  • Oncogene Proteins, Fusion
  • Phosphates
  • Recombinant Fusion Proteins
  • Fibroblast Growth Factors
  • Fibroblast Growth Factor-23
  • FP-1039
  • Receptor, Fibroblast Growth Factor, Type 1
  • Calcium